HAYA Therapeutics SA, a clinical-stage biotechnology company pioneering precision RNA-guided therapeutics that target the regulatory genome to reprogram disease-driving cell states in common, chronic, and age-related diseases, announced that the first cohort has been fully enrolled and dosed in the company’s Phase 1 clinical trial evaluating HTX-001, its first-in-class long non-coding RNA (lncRNA)-targeting therapy for nonobstructive hypertrophic cardiomyopathy (nHCM).
HTX-001 offers a differentiated and potentially disease-modifying approach to treating patients with nHCM. The investigational molecule is an antisense oligonucleotide designed to downregulate WISPER, a heart stress-specific lncRNA overexpressed in patients with hypertrophic cardiomyopathy, including nHCM. By decreasing WISPER expression in cardiac myofibroblast cells, HTX-001 is intended to promote cell-state reprogramming of this fibrotic and pathological cell population back toward a healthy state. Preclinical studies demonstrate that HTX-001 reduces pathological cardiac fibrosis and improves heart function.
The advancement of HTX-001 into the clinic represents a significant milestone in translating HAYA’s foundational regulatory genome science into a potentially new therapeutic approach. WISPER was first discovered in 2017 by Samir Ounzain, Ph.D., co-founder and CEO of HAYA Therapeutics, who identified its role in cardiac fibrosis and remodeling. Building on this discovery, Dr. Ounzain co-founded HAYA with Daniel Blessing, Ph.D., co-founder and CSO, who has led the research and development team responsible for advancing the tailored design and translation of HTX-001 from scientific concept into clinical development.
